Pharm Exec's 2012 Pipeline Report

Just days before this article went to press, FDA approved the first of a new kind of oral enzyme treatment that mediates cellular response, Incyte/Novartis' Jakafi, for a rare bone marrow disease called myelofibrosis. The next JAK inhibitor, Pfizer's toficitinib, could hit the market late next year, meaning a lot of rheumatoid arthritis patients will never again have to sit in a hospital for a couple of hours to get an anti-TNF infusion. Many innovative drugs, long out of the gate, are closing in on the finish line; science is back, and a better understanding of the way genomics shapes disease is bringing about better outcomes, and sometimes faster approvals. Industry is not off the hook, though, and no one should anticipate a golden era of easy profits. Jobs were lost last year, and R&D facilities shuttered, but consolidation has in some ways reenergized and fortified the industry; organizations have gone back to doing what they do best, or have committed to a new R&D or discovery model, or a new therapeutic class. "I think the financial crisis of the last several years has clarified and concentrated quality in the industry," says Raghuram Selvaraju, senior vice president and senior biotech analyst, Morgan Joseph TriArtisan. "People are focused on doable projects in drug development."

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There are a handful of drugs destined to reach blockbuster status in Pharm Exec's 2012 Pipeline Report, but plenty of other half-billion-dollar drugs that, when strung together, make for a convincing portfolio. Hepatitis C is a hotbed of activity, with Gilead swallowing up Pharmasset and its PSI-7977 candidate, a drug that sidesteps interferon and its side effects. In cancer, companion diagnostics have moved to the foreground, and with them a dramatic change in patient outcomes. Clovis Oncology's CO-101, a first-line treatment for pancreatic cancer, is backed by a Roche-made companion diagnostic. Medivation and Astellas' MDV-3100, a Phase III oral drug for prostate cancer, is likely to be approved at the end of next year, according to Adis R&D Insight, and it could take on breast cancer next.

For very high triglycerides, Amarin's AMR 101, an ultraconcentrated omega-3 fatty acid, is expected to gain approval next July, with huge sales to follow. Several novel antibiotics will go to war with the superbugs, while others will tame pneumonia, the seventh-leading cause of death in the US. In diabetes, longer- and faster-acting agents will make adherence easier on patients while bringing in blockbuster sales. Biogen Idec's BG-12, a Gilenya without the side effects, has the makings of a blockbuster too; it's expected to launch sometime in 2012. Biomarin, an organization committed to rare, orphan diseases, continues to dig out patients around the world for clinical trials on enzyme replacement therapies, drugs that regularly save lives.

Industry knows that it will have to prove a drug's value to get paid—more head-to-head clinical trials are happening all the time—and in the end, that's good for everyone. The organizations that are succeeding are the ones that are in research for the long run—and are acting like it.— Ben Comer, Senior Editor